title: Outcome of patients activating an unrelated donor search for severe acquired aplastic anemia.
abstract: Patients with severe aplastic anemia (SAA) without a sibling donor receive immunosuppressive treatment (IST) with anti-thymocyte globulin (ATG). In the case of no response to IST, a voluntary unrelated donor (VUD) search is usually started. This study analyzes the outcome of ATG-refractory SAA patients activating a VUD search. Of 179 patients, 68 had at least one HLA-A, -B, and -DR matched donor identified and underwent HSCT while 50 also with a donor were not transplanted because of early death (8), late response to IST (34), transplant refusal (1), or other (7). Conversely, 61 had no matched donor, 13 of those ultimately received a mismatched HSCT. All but one received marrow stem cells. Among patients aged <17 years, those with at least one matched donor had a significant higher 4-year survival as compared to others (79% ± 6% versus 53% ± 10%, P = 0.01). There was also a survival advantage independent of recipient age when the donor search was initiated in the recent 2000-2005 study-period (74% ± 6% versus 47% ± 10%, P < 0.05). In multivariate analysis, the identification of a matched VUD tended to impact favourably on survival in patients with a recent donor search (P = 0.07). This study provides evidence for the use of unrelated donor HSCT in children and adults with IST-refractory SAA.
authors.affiliation: Henri Mondor Hospital, Créteil, France.
authors.forename: Sébastien
authors.initials: S
authors.lastname: Maury
authors.forename: Marie-Lorraine
authors.initials: ML
authors.lastname: Balère-Appert
authors.forename: Simona
authors.initials: S
authors.lastname: Pollichieni
authors.forename: Rosi
authors.initials: R
authors.lastname: Oneto
authors.forename: Ibrahim
authors.initials: I
authors.lastname: Yakoub-Agha
authors.forename: Franco
authors.initials: F
authors.lastname: Locatelli
authors.forename: Jean-Hugues
authors.initials: JH
authors.lastname: Dalle
authors.forename: Edoardo
authors.initials: E
authors.lastname: Lanino
authors.forename: Alain
authors.initials: A
authors.lastname: Fischer
authors.forename: Andrea
authors.initials: A
authors.lastname: Pession
authors.forename: Anne
authors.initials: A
authors.lastname: Huynh
authors.forename: Walter
authors.initials: W
authors.lastname: Barberi
authors.forename: Mohamad
authors.initials: M
authors.lastname: Mohty
authors.forename: Antonio
authors.initials: A
authors.lastname: Risitano
authors.forename: Noel
authors.initials: N
authors.lastname: Milpied
authors.forename: Gérard
authors.initials: G
authors.lastname: Socié
authors.forename: Andrea
authors.initials: A
authors.lastname: Bacigalupo
authors.forename: Judith
authors.initials: J
authors.lastname: Marsh
authors.forename: Jakob R
authors.initials: JR
authors.lastname: Passweg
mesh_terms: D000328:Adult; D000367:Age Factors; D000741:Anemia, Aplastic / Q000401:mortality / Q000628:therapy; D000961:Antilymphocyte Serum / Q000008:administration & dosage*; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D046148:Donor Selection; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006650:Histocompatibility Testing; D006801:Humans; D007166:Immunosuppressive Agents / Q000008:administration & dosage*; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D008875:Middle Aged; D012189:Retrospective Studies; D012720:Severity of Illness Index; D015996:Survival Rate; D014184:Transplantation, Homologous; D061349:Unrelated Donors
chemical_list: D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents